Cardiomyopathy: TTR Amyloid: efficacy and safety of acoramidis in transthyretin amyloid cardiomyopathy - ATTRibute-CM Trial, ESC Meeting August 2023

The ATTRibute-CM trial was a randomized, double-blind, placebo-controlled trial that evaluated the efficacy and safety of acoramidis in patients with transthyretin amyloid cardiomyopathy (ATTR-CM). The trial enrolled 632 patients with symptomatic ATTR-CM. Patients were randomly assigned to receive either acoramidis 800 mg twice daily or a placebo for 30 months.

The trial's primary endpoint was a hierarchical analysis of all-cause mortality, cardiovascular-related hospitalization, NT-proBNP, and 6-minute walk distance. The trial found that acoramidis was associated with a number of benefits compared to placebo, including:

• A 25% reduction in all-cause mortality
• A 50% reduction in cardiovascular-related hospitalizations
• A 39.64-meter improvement in 6-minute walk distance
• A 9.94-point improvement in KCCQ-OS score
• A 7.1 mg/dL increase in serum transthyretin levels

Acoramidis was generally well-tolerated. The most common side effects were nausea, vomiting, and diarrhea.

The results of the ATTRibute-CM trial suggest that acoramidis is an effective and safe treatment for ATTR-CM. Acoramidis is a potential alternative to tafamidis, the only other FDA-approved drug tafamidis for treating ATTR-CM.

Here are some additional details about the ATTRibute-CM trial:

• The trial was conducted in 40 countries.
• The median age of the participants was 78 years.
• The majority of the participants were male (90%).
• The most common ATTR-CM subtype was wild-type (80%).
• The trial was funded by Akcea Therapeutics.

The results of the ATTRibute-CM trial are a major step forward in the treatment of ATTR-CM. Acoramidis offers a new option for patients with this serious condition, and it is likely to be an important treatment option for many years to come.